Campaign for Access to Treatments for Spinal Muscular Atrophy

In infants with spinal muscular atrophy (SMA), treatment with Spinraza (nusinersen), the first FDA-approved drug for SMA, reduces the risk of death or permanent ventilation, according to new results from the Phase 3 ENDEAR study (NCT02193074).

The evaluation of medicines in the EU can be through single national submissions (in the first instance then a mutual review lead by the country who made the first assessment) but in the case of medicines of high importance like…